EAST HANOVER, N.J., July 8, 2011 /PRNewswire/ — Novartis
Pharmaceuticals Corporation (“Novartis”) announced today Phase III
trial results that showed more than one-third of patients taking
Afinitor® (everolimus) tablets* experienced a 50% or greater
reduction in the size of their subependymal giant cell astrocytomas
(SEGAs), non-cancerous brain tumors associated with tuberous
sclerosis complex (TSC)(1,2,6). This study, the largest prospective
clinical trial to date in this patient population, is being
presented on Saturday, July 9 at the International TSC Research
Conference in Washington, D.C.
Currently, Afinitor is approved in the US for the following
indication: to treat patients with SEGA associated with tuberous
sclerosis who require therapeutic intervention but are not
candidates for curative surgical intervention. The effectiveness of
everolimus is based on an analysis of change in SEGA volume.
Additionally, the indication states, clinical benefit such as
improvement in disease-related symptoms or increase in overall
survival has not been shown(7).
Tuberous sclerosis complex affects approximately 25,000 to
40,000 people in the US and one to two million people worldwide,
and is associated with a variety of resulting disorders including
seizures, swelling in the brain (hydrocephalus), developmental
delays and skin lesions(2,6). Also known as tuberous sclerosis
(TS), TSC is a genetic disorder that may cause non-cancerous tumors
to form in vital organs and can affect many different parts of the
body, most commonly the brain and kidney(6,8). Signs and symptoms
of TSC vary depending on which system and which organs are
involved(6). SEGAs occur in up to 20% of patients with TSC. In
countries where everolimus is not approved, brain surgery is the
only treatment option for patients with growing SEGAs(2).
The 117-patient, randomized, placebo-controlled Phase III
EXIST-1 (EXamining everolimus In a Study of TSC) trial met its